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Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Centre Hospitalier Universitaire, Amiens
ClinicalTrials.gov Identifier:
NCT01441557
First received: September 26, 2011
Last updated: June 22, 2012
Last verified: June 2012
  Purpose

Main objectives:

Evaluate the effectiveness of an administration of 3,4-diaminopyridine (FIRDAPSE ®) in severe botulinic poisoning in measuring the effect on electrophysiological and respiratory parameters

Secondary Objective:

Study the natural history of electrophysiological and respiratory parameters during the botulinic intoxication

Primary endpoint:

Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.

Study Design:

Pilot study, prospective, interventional.

Study population:

Case series (n = 8 patients) suffering from botulinic type A, respiratory failure, but with no other organ failure

Experimental treatment :

3,4-diaminopyridine, FIRDAPSE ® (BioMarin) The dosage will be gradually increased according to a predetermined scheme and will not exceed 60 mg / day and 20 mg / dose.

Statistics:

Intra-individual comparison of physiological parameters measured before and after administration of 3,4-diaminopyridine. Electromyographic and respiratory parameters will be measured for each patient. Then a dose of 10 mg of 3,4-diaminopyridine will be administrated. If this dose is well tolerated and provides a relative improvement of 10% for at least one of the parameters studied, the dose will be maintained at 10 mg for 48 hours 3 times a day then increased to 20 mg.

The primary endpoint is the change in the amplitude of muscle response evaluated by the subtraction of amplitude at T1.5 and T0.


Condition Intervention Phase
Botulism
Drug: 3,4-diaminopyridine
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: Pilot Study on the Usefulness of 3,4-diaminopyridine in the Treatment of Botulism

Resource links provided by NLM:


Further study details as provided by Centre Hospitalier Universitaire, Amiens:

Primary Outcome Measures:
  • electrophysiological [ Time Frame: 90 min ] [ Designated as safety issue: No ]

    Primary endpoint:

    Clinical, electrophysiological and respiratory before and after administration of 3,4-diaminopyridine.



Enrollment: 3
Study Start Date: September 2011
Study Completion Date: March 2012
Primary Completion Date: February 2012 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: 3,4-diaminopyridine
    test dose 10 mg then 20 mg, then depending on tolerance and efficacy full-dose administration(from 10mg /8h to 20 mg /8h)
  Show Detailed Description

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • botulism
  • ventilation

Exclusion Criteria:

  • renal failure
  • cardiac failure
  • pregnancy
  • under age 18
  • hepatic failure
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01441557

Locations
France
CHU
Amiens, France, 80000
Sponsors and Collaborators
Centre Hospitalier Universitaire, Amiens
Investigators
Principal Investigator: Herve DUPONT, Md, PhD CHU Amiens
  More Information

No publications provided

Responsible Party: Centre Hospitalier Universitaire, Amiens
ClinicalTrials.gov Identifier: NCT01441557     History of Changes
Other Study ID Numbers: 2011-004656-19
Study First Received: September 26, 2011
Last Updated: June 22, 2012
Health Authority: France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)

Keywords provided by Centre Hospitalier Universitaire, Amiens:
mechanical ventilation

Additional relevant MeSH terms:
Botulism
Bacterial Infections
Chemically-Induced Disorders
Clostridium Infections
Foodborne Diseases
Gram-Positive Bacterial Infections
Nervous System Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Neurotoxicity Syndromes
Poisoning

ClinicalTrials.gov processed this record on November 24, 2014